International Journal of Technology Assessment in Health Care最新文献

Background: International development agendas increasingly push for access to healthcare for all through universal healthcare coverage. Health economic evaluations and health technology assessment (HTA) could provide evidence to support this but do not routinely incorporate consideration of equitable access.

Methods: We undertook an international scoping review of health economic evaluation and HTA guidelines to examine how well issues of healthcare access and equity are represented, evidence recommendations, and gaps in current guidance to support evidence generation in this area. Guidelines were sourced from guideline repositories and websites of international agencies and organizations providing best practice methods guidance. Articles providing methods guidance for the conduct of HTA, or health economic evaluation, were included, except where they were not available in English and a suitable translation could not be obtained.

Results: The search yielded forty-seven national, four international, and nine independent guidelines, along with eighty-six articles providing specific methods guidance. The inclusion of equity and access considerations in current guidance is extremely limited. Where they do feature, detail on specific methods for providing evidence on these issues is sparse.

Discussion: Economic evaluation could be a valuable tool to provide evidence for the best healthcare strategies that not only maximize health but also ensure equitable access to care for all. Such evidence would be invaluable in supporting progress towards universal healthcare coverage. Clear guidance is required to ensure evaluations provide evidence on the best strategies to support equitable access to healthcare, but such guidance rarely exists in current best practice and guidance documents.

Objectives: Healthcare disinvestment requires multi-level decision-making, and early stakeholder engagement is essential to facilitate implementation and acceptance. This study aimed to explore the perceptions of Malaysian healthcare stakeholders to disinvestment initiatives as well as identify disinvestment activities in the country.

Methods: A cross-sectional online survey was conducted from February to March 2023 among Malaysian healthcare stakeholders involved in resource allocation and decision-making at various levels of governance. Response frequencies were analyzed descriptively and cross-tabulation was performed for specific questions to compare the responses of different groups of stakeholders. For free-text replies, content analysis was used with each verbatim response examined and assigned a theme.

Results: A total of 153 complete responses were analyzed and approximately 37 percent of participants had prior involvement in disinvestment initiatives. Clinical effectiveness and cost-effectiveness ranked as the most important criteria in assessment for disinvestment. Surprisingly, equity was rated the lowest priority despite its crucial role in healthcare decision-making. Almost 90 percent of the respondents concurred that a formal disinvestment framework is necessary and the importance of training for the program's successful implementation. Key obstacles to the adoption of disinvestment include insufficient stakeholder support and political will as well as a lack of expertise in executing the process.

Conclusions: While disinvestment is perceived as a priority for efficient resource allocation in Malaysian healthcare, there is a lack of a systematic framework for its implementation. Future research should prioritize methodological analysis in healthcare disinvestment and strategies for integrating equity considerations in evaluating disinvestment candidates.

Objectives: The primary objectives of this umbrella review were to (a) quantify the relative importance, of "severity" and "rarity" criteria in health resource allocation; and (b) analyze the contextual factors influencing the relative importance. The secondary objective was to examine how "severity" and "rarity" criteria are defined.

Methods: Searches were carried out in PubMed and Embase to identify eligible systematic reviews. Quality appraisal of systematic reviews was undertaken. From identified systematic reviews, primary studies were extracted and further screened for eligibility. The inclusion of severity and rarity criteria and their respective weights in primary studies were examined. Descriptive and regression analyses were performed.

Results: Twenty-nine systematic reviews were screened, of which nine met the inclusion criteria. Primary studies included in these systematic reviews were retrieved and screened, resulting in forty articles included in the final analysis. Disease severity was more frequently considered (n = 29/40) than disease rarity (n = 23/40) as an evaluation criterion. Out of all cases where both were included as evaluation criteria, disease severity was assigned higher weights 84 percent of the time (n = 21/25).

Conclusions: Our review found consistent evidence that disease severity is more relevant and preferred to rarity as a priority-setting criterion albeit constraints in statistical analysis imposed by limited sample size and data availability. Where funding for rare diseases is concerned, we advocate that decision-makers be explicit in clarifying the significance of disease severity and/or rarity as a value driver behind decisions. Our findings also reinforce the relevance of disease severity as a criterion in priority setting.

Objectives: To describe the type of evidence and the clinical benefit of cancer medicines assessed for funding in Australia by the Pharmaceutical Benefits Advisory Committee (PBAC) and to assess it with the European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS).

Methods: All data on applications submitted to PBAC between 2010 and 2020 were extracted from PBAC Public Summary Documents available online. ESMO-MCBS ratings were retrieved from the ESMO-MCBS website.

Results: Then, 182 cancer indications for 100 cancer medicines were examined by PBAC, including 124 (68.1 percent) for solid tumors and 58 (31.9 percent) for hematological cancers. A total of 137 (75.3 percent) indications were recommended for PBS funding and 40 (21.9 percent) were rejected. Randomized clinical trials (RCTs) were the main source of evidence in 154 indications (84.6 percent), single-arm studies in 28 (15.4 percent) indications. Statistically significant improvement in overall survival (OS) was reported in 80 (44 percent) of the indications, with a median OS gain of 3.0 months (range 0.9-17.0) for solid tumors and 8.2 months (range 1-49.1) for hematological cancers when mature OS data were available. The ESMO-MCBS score was available for 99 solid tumor indications, of which 51 (51.5 percent) showed substantial clinical benefit according to ESMO-MCBS, including 40 (54.1 percent) of PBAC-recommended indications and 9 (42.9 percent) of PBAC-rejected indications. There was no association between the ESMO scoring and PBAC decision.

Conclusions: Most cancer medicines indications considered by PBAC were supported by RCTs. A minority showed a substantial improvement in OS.

Background: Health technology assessment (HTA) organizations generate guidelines to inform healthcare practices toward improved health outcomes. This review sought to identify and classify outcomes of guidelines from HTA organizations within published research.

Methodology: We performed a systematic mixed studies review of empirical studies that (a) referred to a published guideline from an HTA organization and (b) reported an outcome resulting from a guideline. We searched the published literature in English or French within seven databases. Outcome types were classified within five dimensions of an existing framework for online health information (e.g., relevance, cognitive/affective impact, and use). Subdimensions were inductively developed. A two-phase sequential data synthesis was performed. Phase 1: a hybrid deductive-inductive thematic analysis identified the types of outcomes and displayed their relationships on a concept map. Phase 2: descriptive statistics were tabulated by the type of outcome.

Results: A total of 6,719 records were retrieved through searches on 6 February 2023. After screening, we included 120 observational studies (twenty-one qualitative, ninety-four quantitative, and five mixed methods). Phase 1 identified twenty-nine types of outcomes. The most frequently reported outcomes were within the organizational dimension (reported in ninety-four studies). The most common subdimensions were "Referrals" (thirty-eight occurrences), the "Quality of Prescriptions" (fifteen occurrences), and the "Quality of Diagnosis" (eight occurrences). For Phase 2, we could only generate descriptive statistics on seventeen outcomes. These were almost equally distributed among positive, neutral, and negative effects. Our results contribute to knowledge about the outcomes of HTA guidelines and options for documenting and measuring them in future evaluations.

Objectives: Health Technology Assessment (HTA) practitioners recognize the significance of qualitative methodologies that focus on how a technology is feasible, meaningfulness, acceptable, and equitable. This mapping aimed to delineate the frameworks employed to synthesize qualitative evidence and assess the quality of synthesis in HTA .

Methods: Mapping was conducted using Medline, LILACS, CINAHL, Embase, Web of Science, Scopus, PsycINFO, Cochrane Library, JBI, and ScienceDirect databases. Gray literature searches included PROQUEST, Open Grey, Canadian Agency for Drugs and Technologies in Health's Grey Matters, Google Scholar, and HTA agency websites. The inclusion criteria were centered on global qualitative evidence synthesis frameworks. The data are presented in the tables.

Results: Of the 2054 articles, 31 were included, mostly from Europe. Guide was the type of document more cited, and most authors are from HTA agencies and universities. Incorporating both patient and family perspectives is the most cited reason for include qualitative evidence. Regardless of the framework or tool, SPICE was the main acronym, and RETREAT was preferred for approach selection. Thematic synthesis dominated analytic methods, and CASP was the primary quality appraisal tool. GRADE-CERQual graded evidence synthesis, with ENTREQ as the top reporting guidance. The GRADE evidence-to-decision framework was mentioned for recommendations.

Conclusion: This mapping highlights the movement incorporate qualitative evidence in HTA employing specific frameworks. Despite the similarities among documents, most of them describe part of the process to synthesize qualitative evidence. Standardizing procedures to incorporate qualitative evidence into HTA can enhance decision-making. These findings offer essential considerations for HTA practice.

Objectives: Evaluating the impact of patient involvement in health technology assessments (HTA) may help improve practices and avoid ineffective activities. Evaluation, however, continues to be infrequent, inconsistent, and often only relates to process quantity or quality. The Patient and Citizen Involvement in HTA Interest Group (PCIG) within Health Technology Assessment International set out to contextualize this impact to support evaluation.

Methods: Given the lack of established methodology to measure impact, the team performed a qualitative analysis of first-hand accounts about perceived changes in HTA due to involvement of patient stakeholders. A questionnaire was developed, piloted, and rolled out to collect personal perspectives from stakeholders with relevant experience. The stories were analyzed in the aggregate to identify themes in the data.

Results: From January 2019 to September 2021, twenty-four responses were collected through PCIG's network. Responses (including one joint industry-HTA body submission) came from patient representatives (12), HTA bodies (11), and industry representatives (2) from North America (5), South America (3), Europe (13), and Asia Pacific (3). Based on themes commonly reported, a three-domain framework for evaluating impact is proposed: impact on basis of HTA result or recommendation, impact on HTA body, and impact on patient participants. The framework includes components under each domain to support reporting.

Conclusions: Using the Three-Domain Impact Framework may be useful in identifying, evaluating, and communicating the value of patient involvement in HTA. Enhancing and increasing reporting practices may improve transparency and facilitate process improvements for meaningful integration of patient stakeholders into HTA appraisals across jurisdictions.

Objectives: A conceptual framework, called Innovation of Health Technology Assessment Methods (IHTAM), has been developed to facilitate the understanding of how to innovate methods of health technology assessment (HTA). However, the framework applicability has not been evaluated in practice. Hence, we aimed to explore framework applicability in three cases of method innovation that are part of the HTx project and to develop a roadmap to improve framework applicability.

Methods: The IHTAM framework was applied to three cases of innovating HTA methods. We collected feedback from case study leaders and consortium members after a training session, an approximately 1-year follow-up of periodic case study meetings, and a general assembly meeting where innovation progresses of the three cases were reported through surveys and interviews. Feedback was then summarized using an open-coding technique.

Results: According to feedback, the framework provided a structured way of deliberation and helped to improve collaboration among HTA stakeholders. However, framework applicability could be improved if it was complemented by a roadmap with a loop structure to provide tailored guidance for different cases, and with items to elaborate actions to be taken by stakeholders. Accordingly, a 48-item roadmap was developed.

Conclusions: The IHTAM framework was generally applicable to the three case studies. A roadmap, with loop structure and actionable items, could complement the framework, and may provide HTA stakeholders with tailored guidance on developing new methods. To further examine the framework applicability, we recommend stakeholders to apply the IHTAM framework and its roadmap in future practice.

Introduction: The costs of low back pain (LBP) are complex and difficult to estimate. This study aims to adapt the Cost for Patients Questionnaire (CoPaQ) for use in LBP populations.

Materials and methods: In a cross-sectional qualitative study, we conducted cognitive interviews to assess the CoPaQ's suitability for addressing costs related to LBP. Three groups of participants were included (n = 5 each): (i) persons with a history of LBP or primary caregiver, (ii) researchers with expertise in LBP, and (iii) primary care providers specialized in treating LBP. The interpretation, analysis, and summary of results used Knafl et al.'s qualitative content analysis method.

Results: Persons with a history of LBP (n = 5), had a median age of 60 years (Interquartile Range (IQR): 26-71.5), and varying durations of LBP, the median duration of LBP 7 years (IQR: 4-32.5). Researchers (n = 5) had a median age of 33 years (IQR: 29-45). Primary care providers (n = 5) had a median age of 40 years (IQR: 37.5-65), and a background in chiropractic care (n = 3) and physiotherapy (n = 2). Content analysis of the interviews revealed sources of error with five pre-determined themes (clarity/comprehension, relevance, inadequate response definition, reference point, perspective modifiers) and one developed theme (organization). We modified the questionnaire for LBP populations based on the feedback.

Conclusion: Our study evaluated the content validity of a questionnaire that assesses the direct and indirect costs associated with LBP. Future studies should pilot this questionnaire with persons of varying LBP severity and compare it with cost diaries.

In many countries, the economics domain forms a routine part of health technology assessments (HTA) next to analyzing the comparative effectiveness and safety of a technology. The method applied most often is economic evaluation, such as cost-effectiveness analysis, which is supposed to support the efficient use of resources. In Austria, economic evaluation has played a negligible role in HTA and reimbursement decisions, even though the country faces the same public healthcare sustainability challenges as others. In this commentary, we argue that while health economics will need to play a more active role in HTA-related decision support to deal with those challenges, current approaches in other countries may have to be broadened to fit the Austrian context. We are outlining four arguments to underpin this perspective: First, economic evaluations (in their current form) are of limited benefit for supporting reimbursement decisions of new high-priced technologies. Second, a broader variety of health economic methods is needed to address the scope of technologies. Third, applying health economic methods requires a reflection on their underlying values. Finally, health economics within HTA needs to go beyond microeconomic analysis of interventions. We are suggesting several alternative methods and approaches, encouraging out-of-the-box thinking and experimenting with methods developed in the academic context but rarely applied in routine HTA. Although some of our topics are unique to Austria, others may equally apply to other healthcare systems. With our thoughts, we aim to stimulate discussions for further developing health economics within HTA in Austria and internationally.